Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1

NCT00865644 | Completed Phase 1 DMC

• 1 other identifier: 08-347
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if imiquimod cream can reverse the growth of neurofibromas. Imiquimod is a skin cream that works by stimulating the body's immune system to respond to tumors. Imiquimod cream is approved for use in patients with various skin lesions, including actinic keratosis, superficial basal cell carcinoma, and external genital warts. Information from these studies, as well as previous laboratory studies, suggest that imiquimod cream may help shrink neurofibromas or keep them from growing.

Conditions

Neurofibromatosis Type 1; Cutaneous Neurofibromas

Keywords

imiquimod 5% cream

Outcome Measures

Primary Outcomes (1)

• To determine the effect of topical imiquimod 5% cream on tumor volume of cutaneous neurofibromas in adult subjects with neurofibromatosis 1

  2 years

Secondary Outcomes (1)

• To correlate the inflammatory infiltrate adjacent to treated lesions during treatment with tumor response and to determine the number of circulating Tregs in this patient population

  2 years

Study Arms (1)

Imiquimod

EXPERIMENTAL

Drug: Imiquimod 5% Cream

Interventions

Imiquimod 5% Cream DRUG

Applied topically to three tumors 5 times per week for a full 6 weeks

Imiquimod

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have a diagnosis of NF1 based on NIH criterial with two or more of the following characteristics:
• six or more cafe-au-lait macules (1.5cm or greater in size)
• skin fold freckling in the axilla or groin
• optic pathway glioma
• two or more Lisch nodules of the iris
• distinctive bony lesions such as dysplasia of the sphenoid wing or of a long bone such as the tibia
• two or more neurofibromas of any type of 1 or more plexiform neurofibroma
• first degree relative with NF1
• Participants must have at least four cutaneous neurofibromas on skin exam with the following qualities:
• the lesion must be discrete by clinical exam and must not be contact with another skin tumor
• the lesion must be amenable to measurement with calipers with minimum dimension of 5mm and maximum dimension of 30mm
• the lesions must be located on the trunk, neck, or extremities (excluding the hands and feet) and must be located in an area that can be photographed
• histologic confirmation of tumor type is not required in the setting of compatible clinical setting
• No treatment with an investigation agent for cutaneous neurofibromas within the last 3 months
• years of age or older

You may not qualify if:

• Pregnant and nursing women
• Patients who have had chemotherapy or radiotherapy within 6 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 6 weeks earlier
• Patients may not be receiving any other investigational agents
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to imiquimod
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Imiquimod

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Aminoquinolines; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Scott R. Plotkin, MD, PhD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: March 17, 2009
• First Posted: March 19, 2009
• Study Start: March 1, 2009
• Primary Completion: April 1, 2010
• Study Completion: December 1, 2013
• Last Updated: January 10, 2017

Record last verified: 2017-01

Locations

US (1)